P1682: health related quality of life in children and adolescents with sickle cell disease: are we sure to get it right? patients and parents might have different opinions.

Topic: 35. Quality of life and palliative care Background: Sickle cell disease (SCD) is a complex chronic and multiorgan genetic disorder of hemoglobin. In addition, SCD is responsible for reduced health-related quality of life (HRQOL). Despite the improvement in the clinical care of patients with SCD, vaso-occlusive crises (VOC), recurrent admissions, and acute illness aid in poor HRQOL. HRQOL is a multifactorial dimension influenced by chronic complications and psychological, social, mental, and economic variables. Aims: The aim was to perceive the HRQOL of children, adolescents and young adults with SCD referred to the Sickle Cell Disease Reference Center, in Padova, Italy. The secondary aim was to relate HRQOL to demographic, clinical, and hematological variables. Methods: We used the Patient-Reported Outcomes Measurement Information System (PROMIS) to assess HRQOL in patients aged 6-26 years: PROMIS Pediatric Profile 49 v.2.0, PROMIS Adult Profile 57 v.2.1, PROMIS Parent Proxy Profile 49 v2.0, PROMIS Emotional Support, PROMIS Instrumental Support, completed in a paper format (several domains were evaluated: Pain Interference, Pain Intensity, Fatigue, Anxiety, Depression, Physical Abilities, Sleep Disorders, Peer Relationship, Family Relationship, Emotional Support, and Instrumental Support). T-Score was obtained through the HealthMeasures Scoring Service platform for each patient, and each domain. A questionnaire to evaluate social and economic variables was also administered. The t-Student Test, the Wilcoxon Test, the Spearman’s Agreement Test, and the Correlation Agreement Test were used to evaluate correlation of PROMIS scores with hematological and clinical variables. Values with p < 0.05 were considered statistically significant. Results: The study involved 53 patients aged 6-26 years (45% female, mean age 14.2 years, 80% HbSS genotype, 83% from Africa), and one parent for patient. 50% of the patients had moderate compromission of HRQOL in at least three PROMIS domains. Two out of three patients experience pain in the last seven days. Patients of 14-26 years had worse symptoms than patients of 6-13 years in all domains, especially in pain interference (p=0,0001), pain intensity (p=0,0039), depression (p=0,0273), and fatigue (p=0.0298). Parents had a low agreement with patients in all domains, especially in anxiety (Figure 1A), and interference of pain (Figure 1B). In fact, parents have an opposite pain interference perception compared to patients, with a negative agreement coefficient (-0.074; Figure 1C). Significantly, none of the young adults (18-26 years) reported receiving adequate emotional support, all were in the range of intermediate-low (Figure 1D); all requested more psychological support and many of them would appreciate tutoring to access the job market. There was a close correlation between sleep disturbances and the number of hospitalizations (p=0,0034). Summary/Conclusion: Our study demonstrated the applicability of PROMIS questionnaires in a cohort of patients with SCD living in Italy, and the reduction in a significant percentage of HRQOL-related domains. HRQOL needs to be better investigated in children and adolescents who have a different perception of disease compared to their parents’ opinion. Young adults need more support for disease management, both practical and psychological.Keywords: Pediatric, Quality of life, Sickle cell patient, Sickle cell disease